R&D pipeline

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.

We’re investigating other potential small molecule medicines targeting the underlying cause of CF. In people with certain types of mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the CFTR protein is not processed and cannot move through the cell normally. This results in little to no protein at the cell surface. Vanzacaftor, formerly known as VX-121, and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the CFTR protein. Deutivacaftor is a potentiator designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. The triple combination of vanzacaftor/tezacaftor/deutivacaftor is being developed as an investigational once-daily treatment for people with CF with certain mutations in the CFTR gene.

For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.

We are investigating VX-522, a CFTR mRNA that can be delivered to the lung by lipid nanoparticles to address the underlying cause of CF lung disease in approximately 5,000 people with CF who do not make any CFTR protein that responds to a CFTR modulator therapy.

For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov.

We’re investigating a portfolio of other small molecule medicines targeting the underlying cause of cystic fibrosis with the aim of achieving carrier levels of CFTR function. This includes CFTR potentiators, which are designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across cell membranes, helping to hydrate and clear mucus from the airways. We’re also researching additional small molecules to address the trafficking and processing defect of the CFTR protein to enable it to move through cells and reach the surface.

We’re investigating potential treatments for people with cystic fibrosis who do not make any CFTR protein. We are investing in our own science and with our external partners CRISPR, Arbor and Moderna to develop other potential approaches with an aim to treat approximately 5,000 people who do not make any CFTR protein that responds to a CFTR modulator therapy. Our efforts to discover and develop therapies to reach as many people as possible with cystic fibrosis through alternative investigational technologies, like mRNA therapies, have been underway for years.

We continue to discover, research and develop a portfolio of small molecule medicines as potential non-opioid medicines for the treatment of both acute and neuropathic pain. Our approach is to inhibit specific channels validated by human biology with the aim of alleviating pain.

We continue to discover, research and develop a portfolio of small molecule medicines as potential non-opioid medicines for the treatment of both acute and neuropathic pain. Our approach is to inhibit specific channels validated by human biology with the aim of alleviating pain.  

We are submitting regulatory filings globally for geographic expansion. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov. 

We’re investigating small molecule medicines aimed at the underlying cause of sickle cell disease.

We are submitting regulatory filings globally for geographic expansion. For information about ongoing clinical studies in the U.S., visit the clinical trials website. For information about non-U.S. sites, visit clinicaltrials.gov. 

We’re investigating small molecule medicines aimed at the underlying cause of transfusion-dependent beta thalassemia.  

We continue to research a portfolio of small molecules as potential medicines for the treatment of alpha-1 antitrypsin deficiency.

We are investigating inaxaplin, formerly known as VX-147, aimed at inhibiting high-risk variants of  APOL1. 

In addition to investigating candidate medicine inaxaplin, formerly known as VX-147, we continue to research and develop a portfolio of small molecule inhibitors for the potential treatment of APOL1-mediated kidney disease.

We are investigating VX-407 for the treatment of ADPKD in patients with a subset of PKD1 genetic variants, with the aim of restoring function to the polycystin 1 (PC1) protein.

We are investigating povetacicept (ALPN-303) with the goal of targeting the underlying cause of IgA nephropathy (IgAN).

We are investigating a novel approach to treating Duchenne muscular dystrophy by delivering CRISPR/Cas9 gene-editing technology to muscle cells to achieve precise changes in the targeted DNA sequence. Specifically, we’re researching CRISPR/Cas9 gene-editing technology with the goal of restoring near-full length dystrophin protein expression by targeting certain mutations in the dystrophin gene that cause the disease. Due to the number of mutations that can cause Duchenne muscular dystrophy, our research consists of multiple different gene-editing programs to potentially address many of the disease-causing mutations.

We are investigating VX-670, an oligonucleotide approach using an intracellular Endosomal Escape Vehicle (EEV™) aimed at targeting the underlying cause of myotonic dystrophy type 1.

VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. It is being evaluated for patients who have type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 is being investigated as an infusion into the hepatic portal vein and requires immunosuppressive therapy to protect the islet cells from immune rejection.

VX-264 is an investigational approach encapsulating cells in a protective device to be surgically implanted in the body. VX-264 is being evaluated without the use of immunosuppressive therapy as the devices are designed to shield the cells from the body's immune system.

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.